Roche’s ef­fort to in­tro­duce its blood can­cer drug Gazy­va to the im­munol­o­gy space just got a boost from the FDA.

Reg­u­la­tors have grant­ed break­through des­ig­na­tion to the use of the CD20-tar­get­ing an­ti­body in lu­pus nephri­tis, a life-threat­en­ing man­i­fes­ta­tion of sys­temic lu­pus ery­the­mato­sus in the kid­ney. Up to 60% of all peo­ple with lu­pus — dis­pro­por­tion­ate­ly women — de­vel­op such in­flam­ma­tion in the kid­ney, the Lu­pus Foun­da­tion of Amer­i­ca es­ti­mates.

San­dra Horn­ing

Im­muno­sup­pres­sants such as my­cophe­no­late mofetil or my­cophe­no­lic acid and cor­ti­cos­teroids are the cur­rent stan­dard of care, but Roche CMO San­dra Horn­ing not­ed that new treat­ment op­tions are need­ed. In a Phase II study, in­ves­ti­ga­tors showed that adding Gazy­va to the reg­i­men helped a larg­er pro­por­tion of pa­tients achieve com­plete re­nal re­sponse at one year.

A Phase III tri­al is slat­ed to be­gin next year.

While Gazy­va has pre­vi­ous­ly been po­si­tioned as a po­ten­tial suc­ces­sor to the ag­ing Rit­ux­an fran­chise, Roche re­cent­ly high­light­ed its po­ten­tial in lu­pus on its Phar­ma Day ear­li­er this week.

The ther­a­peu­tic piv­ot is part of a larg­er at­tempt to re­duce Roche’s de­pen­dence on the es­tab­lished — and high­ly prof­itable — on­col­o­gy port­fo­lio in fa­vor of nov­el drugs and tar­gets, Cowen an­a­lyst Steve Scala wrote in a note.

Roche made clear that mar­gins were not suf­fer­ing through the launch of new growth dri­vers Ocre­vus (mul­ti­ple scle­ro­sis) and Hem­li­bra (he­mo­phil­ia). Ro­bust up­take of both drugs has buoyed the topline while new pro­duc­tiv­i­ty ini­tia­tives saved costs, ex­em­pli­fied by suc­cess­ful launch­es de­spite adding no head count to sales forces. The com­pa­ny hopes to repli­cate this suc­cess when launch­ing in­to NMOSD with satral­izum­ab, IBD with etrolizum­ab, SMA with ris­diplam, Hunt­ing­ton’s with ASO-HTT, and lu­pus with Gazy­va.

The drug has been ap­proved to treat chron­ic lym­pho­cyt­ic leukemia and fol­lic­u­lar lym­phoma, with or fol­low­ing chemo. Its 2018 sales to­taled CHF 390 mil­lion (rough­ly $392 mil­lion).

By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.

Following a successful Phase III study in April showcasing the safety and potential of its new sleep drug, Idorsia posted some mixed news in the second Phase III study, but that won’t stop a planned filing aimed at regulatory approval.

The drug, a dual orexin receptor antagonist (DORA) called daridorexant, was found to significantly improve sleep maintenance and subjective total sleep time in 25 mg doses, replicating results from the first Phase III study. However, improvements in sleep onset and daytime functioning narrowly missed statistical significance, despite numerical consistency with the April study.